We Did It! Now Let’s Do It AGAIN! “$200K for SMA” Campaign Continues

March 01, 2011 by Victoria Strong

You already know that GSFs original "$200K for SMA" goal was far surpassed -- YEEHAW!!! But we aren't stopping there and we're as dedicated and motivated as ever to continue fundraising for promising Spinal Muscular Atrophy research programs! Are you?

So what's next for GSF and the "$200K for SMA" campaign? --> Dr. Kaspar's gene therapy research is extremely promising, but some material work is still ahead. And we remain determined to help take this research out of the lab and to an actual treatment for SMA. So, we are continuing our successful partnership with FightSMA whose 20-year relationship with SMA researchers and strong research legacy make them invaluable in moving forward efficiently and effectively.

While Dr. Kaspar continues to work on safety and efficacy studies that GSF and FightSMA helped fund, we have already begun funding a research program that is necessary to take gene therapy out of the lab and into patients -- a large animal or pre-clinical model -- which does not currently exist. Dr. Monique Lorson began working on a pre-clinical model after attending the American Society of Gene and Cell Therapy meeting -- sponsored by FightSMA. Numerous investigators talked about how the pre-clinical models are needed for therapeutics and with initial funding from FightSMA, Dr. Lorson was able to begin this study and she has already generated a preliminary model. The next venture is generating a true SMA model with two deleted copies of SMN and she needs our help to get it there. And Dr. Kaspar (along with other SMA researchers) is counting on this pre-clinical model to push his SMA research ahead!

Here's what Dr. Kaspar and Dr. Lorson have to say about this important project:

Developing larger animal models for Spinal Muscular Atrophy is needed for research and therapeutic development in SMA. Currently, there are only tissue culture models of SMA and mouse models to screen drugs and therapies. The ability to test a therapy in a larger model of the disease can provide valuable data and provides a path towards translating to humans. Currently, there is no large model of SMA, so we perform safety and toxicology studies in normal animals. The need to have a larger model will give further efficacy and safety confidence in a drug or therapy. I applaud FightSMA for the vision to support the SMA large animal model that is under development in Dr. Monique Lorsons lab. We appreciate the FightSMA and GSF response to this pressing need. -- Dr. Brian Kaspar, PhD, Principal Investigator at Nationwide Children's Hospital

The generation of an SMA animal model could never be realized without the vision and pioneer spirit of FightSMA. FightSMA saw the research potential and believed in the expertise of our research team. Thank you with extreme gratitude. -- Dr. Monique Lorson, PhD, Investigator at University of Missouri Columbia

So what are we asking of YOU? --> Get involved.

How can you get involved > Simple.

Continue to support GSF and FightSMA fundraising efforts.

Start your own fundraiser.

Anything can become a fundraiser. Get creative. Think outside the box. Set a goal. And then go for it. All that we ask is that you:

Set a goal and strive to meet it.

Plan to have all funds raised by the end of September 2011.

Have a blast!

Whether it be running a race or hosting a golf tournament, a letter writing campaign or a garage sale, a birthday wish or a lemonade stand ALL efforts matter and will make a difference for this exciting program! We've proven that before and we'll prove it again. By signing up for the "$200K for SMA" campaign, GSF will help you through the fundraising process and we'll work together to reach the overall $200,000 goal. We all came together and exceeded -- absolutely crushed -- our first "$200K for SMA" goaland we know we can do it again -- together! Contact Victoria Strong (Victoria@theGSF.org) to sign up for the "$200K for SMA" campaign!

100% of your donation to the Gwendolyn Strong Foundation under the "$200K for SMA" campaign will go directly to this exciting research program -- yes, ONE HUNDRED PERCENT. Thank you for making a difference!

"$200K for SMA" > One person and one dollar at a time. Together we can end SMA!